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WO2018192982A3 - Méthodes de production de vecteurs viraux adéno-associés - Google Patents

Méthodes de production de vecteurs viraux adéno-associés Download PDF

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Publication number
WO2018192982A3
WO2018192982A3 PCT/EP2018/059918 EP2018059918W WO2018192982A3 WO 2018192982 A3 WO2018192982 A3 WO 2018192982A3 EP 2018059918 W EP2018059918 W EP 2018059918W WO 2018192982 A3 WO2018192982 A3 WO 2018192982A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
aav
adeno
methods
relates
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PCT/EP2018/059918
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English (en)
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WO2018192982A2 (fr
Inventor
Conrad VINK
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Glaxosmithkline Intellectual Property Development Limited
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from GBGB1706090.6A external-priority patent/GB201706090D0/en
Priority claimed from GBGB1718179.3A external-priority patent/GB201718179D0/en
Application filed by Glaxosmithkline Intellectual Property Development Limited filed Critical Glaxosmithkline Intellectual Property Development Limited
Priority to BR112019021595-6A priority Critical patent/BR112019021595A2/pt
Priority to EP18724467.8A priority patent/EP3612635A2/fr
Priority to JP2019556592A priority patent/JP2020517238A/ja
Priority to CA3058970A priority patent/CA3058970A1/fr
Priority to CN201880040553.6A priority patent/CN110785495A/zh
Publication of WO2018192982A2 publication Critical patent/WO2018192982A2/fr
Publication of WO2018192982A3 publication Critical patent/WO2018192982A3/fr
Priority to JP2023035225A priority patent/JP2023081975A/ja

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

L'invention concerne une cellule productrice de virus adéno-associé (VAA) comprenant des séquences d'acide nucléique codant : un gène de répétition/coiffe; des gènes de virus auxiliaire; et le génome d'ADN de la particule de vecteur de VAA, lesdites séquences d'acide nucléique étant toutes intégrées ensemble au niveau d'un locus unique dans le génome de cellule productrice de VAA. L'invention concerne également des vecteurs d'acide nucléique ayant une origine de réplication non mammifère et la capacité à contenir au moins 25 kilobases (kb) d'ADN, et est caractérisée en ce que ledit vecteur d'acide nucléique comprend des séquences d'acide nucléique codant : un gène de répétition/coiffe, et des gènes de virus auxiliaire. L'invention concerne également des utilisations et des méthodes faisant appel audit vecteur d'acide nucléique afin de produire des lignées cellulaires stables d'encapsidation et de production de VAA.
PCT/EP2018/059918 2017-04-18 2018-04-18 Méthodes de production de vecteurs viraux adéno-associés WO2018192982A2 (fr)

Priority Applications (6)

Application Number Priority Date Filing Date Title
BR112019021595-6A BR112019021595A2 (pt) 2017-04-18 2018-04-18 Célula produtora de vírus adeno-associado, vetor de ácido nucleico, métodos para produzir uma linhagem de células de empacotamento estável de vírus adeno-associado e uma partícula de vetor de vírus adeno-associado com defeito de replicação, célula de empacotamento de vírus adeno-associado, e, partícula de vetor de vírus adeno-associado com defeito de replicação.
EP18724467.8A EP3612635A2 (fr) 2017-04-18 2018-04-18 Méthodes de production de vecteurs viraux adéno-associés
JP2019556592A JP2020517238A (ja) 2017-04-18 2018-04-18 アデノ随伴ウイルスベクターの産生方法
CA3058970A CA3058970A1 (fr) 2017-04-18 2018-04-18 Methodes de production de vecteurs viraux adeno-associes
CN201880040553.6A CN110785495A (zh) 2017-04-18 2018-04-18 腺相关病毒载体的生产方法
JP2023035225A JP2023081975A (ja) 2017-04-18 2023-03-08 アデノ随伴ウイルスベクターの産生方法

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GBGB1706090.6A GB201706090D0 (en) 2017-04-18 2017-04-18 Methods for adeno-associated viral vector production
GB1706090.6 2017-04-18
GB1718179.3 2017-11-02
GBGB1718179.3A GB201718179D0 (en) 2017-11-02 2017-11-02 Adeno-associated viral vector packaging and producer cell lines

Publications (2)

Publication Number Publication Date
WO2018192982A2 WO2018192982A2 (fr) 2018-10-25
WO2018192982A3 true WO2018192982A3 (fr) 2019-03-21

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2018/059918 WO2018192982A2 (fr) 2017-04-18 2018-04-18 Méthodes de production de vecteurs viraux adéno-associés

Country Status (7)

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US (2) US10858631B2 (fr)
EP (1) EP3612635A2 (fr)
JP (2) JP2020517238A (fr)
CN (1) CN110785495A (fr)
BR (1) BR112019021595A2 (fr)
CA (1) CA3058970A1 (fr)
WO (1) WO2018192982A2 (fr)

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SG11202106217TA (en) 2018-12-21 2021-07-29 Lonza Walkersville Inc Adeno-associated virus (aav) producer cell line and related methods
WO2020235543A1 (fr) * 2019-05-20 2020-11-26 Ｊｃｒファーマ株式会社 Molécule d'acide nucléique utilisée pour la production d'un virion aav recombinant
GB202001484D0 (en) * 2020-02-04 2020-03-18 Oxford Genetics Ltd DNA amplification method
US20230159950A1 (en) * 2020-03-17 2023-05-25 Biogen Ma Inc. Methods and compositions for production of recombinant adeno-associated viruses
AU2021319208A1 (en) 2020-07-30 2023-03-02 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
GB202013060D0 (en) * 2020-08-21 2020-10-07 Oxford Genetics Ltd Cell line
EP4242316A4 (fr) * 2020-11-04 2024-09-04 Synplogen Co., Ltd. Plasmide intégratif
EP4293118A4 (fr) * 2021-02-12 2025-01-22 Fujifilm Corp Kit de production de virus adéno-associé, et utilisation dudit kit
WO2022209987A1 (fr) 2021-03-29 2022-10-06 株式会社カネカ Vecteur, procédé de préparation d'adn linéaire fermé à liaison covalente l'utilisant, procédé de préparation de vecteur parvoviral et cellule de production de vecteur parvoviral
EP4351631A4 (fr) * 2021-06-11 2025-04-23 Spark Therapeutics Inc Méthodes de régulation de la production de virus adénoassociés
MX2024004647A (es) 2021-10-18 2024-05-02 Regeneron Pharma Celulas eucariotas que comprenden polinucleotidos de virus asociados a adenovirus.
CN119546751A (zh) * 2022-06-20 2025-02-28 布拉默生物有限责任公司 改进的重组腺相关病毒生产方法
CN116042544A (zh) * 2022-09-01 2023-05-02 中国科学院深圳先进技术研究院 一种血清型13型重组腺相关病毒及其制备方法和应用
CN116200429A (zh) * 2022-09-02 2023-06-02 中国科学院深圳先进技术研究院 一种重组腺相关病毒组合物及其在精准稀疏标记中的应用
CN116064672B (zh) * 2022-09-15 2025-02-14 思鹏生物科技(苏州)有限公司 利用ires基因提升aav包装效率的方法及其功能基因
WO2024160232A1 (fr) * 2023-02-01 2024-08-08 纽福斯(苏州)生物科技有限公司 Système d'emballage de plasmide et son utilisation
CN117683797B (zh) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN117343927B (zh) * 2023-12-06 2024-03-12 上海药明巨诺生物医药研发有限公司 一种工程化细胞的核酸提取方法
CN117660534B (zh) * 2023-12-13 2024-05-07 广州派真生物技术有限公司 一种降低重组腺相关病毒中宿主细胞dna残留的辅助质粒及应用
CN117660532B (zh) * 2023-12-13 2024-08-09 广州派真生物技术有限公司 一种降低重组腺相关病毒中rcAAV残留的辅助质粒及应用

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Also Published As

Publication number Publication date
US10858631B2 (en) 2020-12-08
EP3612635A2 (fr) 2020-02-26
JP2020517238A (ja) 2020-06-18
CN110785495A (zh) 2020-02-11
CA3058970A1 (fr) 2018-10-25
BR112019021595A2 (pt) 2020-05-12
JP2023081975A (ja) 2023-06-13
WO2018192982A2 (fr) 2018-10-25
US20180327722A1 (en) 2018-11-15
US20210062161A1 (en) 2021-03-04

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